Current Research
CURRENT RESEARCH
What we are funding now
On July 24, 2018, we reached a milestone when the first dose of gene therapy was delivered to a GSD patient. The GSD team at the University of Connecticut is leading the study and initial results are extremely positive. Phase 3 of this trial will commence in January 2022.
CRISPR/Cas9- based gene editing system allows researchers to edit DNA sequences and modify gene function. Many scientists believe it is a novel approach to curing genetic disorders. The Children’s Fund for GSD Research is currently funding a study led by Dr. Janice Chou at The National Institute of Health (NIH) to determine if this is a safe and viable option to cure Glycogen Storage Disease.
Hepatocellular adenoma/carcinoma (HCA/HCC) is a long-term complication of GSDIa. Scientists at The University of Connecticut are conducting studies to determine why and the best way to prevent this.
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" ... because every child deserves to be healthy."